1. Gene therapy for lysosomal storage disorders: recent advances for metachromatic leukodystrophy and mucopolysaccaridosis I. Issue 4 (30th May 2017) Authors: Penati, Rachele; Fumagalli, Francesca; Calbi, Valeria; Bernardo, Maria Ester; Aiuti, Alessandro Journal: Journal of inherited metabolic disease Issue: Volume 40:Issue 4(2017) Page Start: 543 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access. Issue 10322 (22nd January 2022) Authors: Fumagalli, Francesca; Calbi, Valeria; Natali Sora, Maria Grazia; Sessa, Maria; Baldoli, Cristina; Rancoita, Paola Maria V; Ciotti, Francesca; Sarzana, Marina; Fraschini, Maddalena; Zambon, Alberto Andrea; Acquati, Serena; Redaelli, Daniela; Attanasio, Vanessa; Miglietta, Simona; De Mattia, Fabiol... Journal: Lancet Issue: Volume 399:Issue 10322(2022) Page Start: 372 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
3. Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study. Issue 5 (May 2019) Authors: Ferrua, Francesca; Cicalese, Maria Pia; Galimberti, Stefania; Giannelli, Stefania; Dionisio, Francesca; Barzaghi, Federica; Migliavacca, Maddalena; Bernardo, Maria Ester; Calbi, Valeria; Assanelli, Andrea Angelo; Facchini, Marcella; Fossati, Claudia; Albertazzi, Elena; Scaramuzza, Samantha; Brigi... Journal: Lancet Issue: Volume 6:Issue 5(2019) Page Start: e239 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
4. Metachromatic leukodystrophy: A single‐center longitudinal study of 45 patients. Issue 5 (4th May 2021) Authors: Fumagalli, Francesca; Zambon, Alberto A.; Rancoita, Paola M. V.; Baldoli, Cristina; Canale, Sabrina; Spiga, Ivana; Medaglini, Stefania; Penati, Rachele; Facchini, Marcella; Ciotti, Francesca; Sarzana, Marina; Lorioli, Laura; Cesani, Martina; Natali Sora, Maria Grazia; Del Carro, Ubaldo; Cugnata, ... Journal: Journal of inherited metabolic disease Issue: Volume 44:Issue 5(2021) Page Start: 1151 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
5. Successful Treatment With Ledipasvir/Sofosbuvir in an Infant With Severe Combined Immunodeficiency Caused by Adenosine Deaminase Deficiency With HCV Allowed Gene Therapy with Strimvelis. Issue 6 (13th November 2018) Authors: Tucci, Francesca; Calbi, Valeria; Barzaghi, Federica; Migliavacca, Maddalena; Ferrua, Francesca; Bernardo, Maria Ester; Canarutto, Daniele; Consiglieri, Giulia; Recupero, Salvatore; Calzatini, Francesco; Gabaldo, Michela; Lucano, Caterina; Casiraghi, Miriam; Darin, Silvia; Dionisio, Francesca; Ma... Journal: Hepatology Issue: Volume 68:Issue 6(2018) Page Start: 2434 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
6. Toward Reference Intervals of ARSA Activity in the Cerebrospinal Fluid: Implication for the Clinical Practice of Metachromatic Leukodystrophy. (10th September 2020) Authors: Morena, Francesco; Argentati, Chiara; Acquati, Serena; DeWall, Stephen; Kelly, Fiona; Calbi, Valeria; Fumagalli, Francesca; Zancan, Stefano; Biffi, Alessandra; Aiuti, Alessandro; Martino, Sabata Journal: Journal of applied laboratory medicine Issue: Volume 6:Number 2(2021) Page Start: 354 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
7. Treatment outcome among children under-five years hospitalized with severe acute malnutrition in St. Mary's hospital Lacor, Northern Uganda. Issue 1 (December 2016) Authors: Nyeko, Richard; Calbi, Valeria; Ssegujja, Boniface; Ayot, Grace Journal: BMC nutrition Issue: Volume 2:Issue 1(2016) Page Start: 1 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
8. Wiskott–Aldrich syndrome: Oral findings and microbiota in children and review of the literature. Issue 1 (24th February 2022) Authors: Lucchese, Alessandra; Cenciarelli, Sabina; Manuelli, Maurizio; Marcolina, Marta; Barzaghi, Federica; Calbi, Valeria; Migliavacca, Maddalena; Bernardo, Maria Ester; Tucci, Francesca; Gallo, Vera; Fraschetta, Federico; Darin, Silvia; Casiraghi, Miriam; Aiuti, Alessandro; Ferrua, Francesca; Cicalese... Journal: Clinical and experimental dental research Issue: Volume 8:Issue 1(2022) Page Start: 28 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗